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High-Dose Eteplirsen Dose Escalation and Randomized Dose-Finding in Duchenne Muscular Dystrophy (Exon 51 Skipping)
2018-001762-42Phase 3Completed
- Sponsor
- Sarepta Therapeutics
- Start
- Jul 2019
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of eteplirsen in Duchenne muscular dystrophy (DMD) (3 arms), sponsored by Sarepta Therapeutics. Primary endpoints: Primary endpoints - open-label dose escalation and Primary endpoints - double-blind dose-finding and comparison. Started Jul 2019; status: completed.
02
Drugs
eteplirsen
03
Conditions
Duchenne muscular dystrophy (DMD)
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